Event Preview: 7th Annual Genome Editing Therapeutics Summit

Cell and Gene Therapy Insights presents an event preview of the 7th Annual Genome Editing Therapeutics Summit. Taking place September 29–30, 2026, in Boston, MA, the summit will unite R&D, preclinical, translational, regulatory, and technical leaders to examine how genome editing can move from a promising modality to a necessary treatment option for patients facing genetically complex diseases.

From proof of concept to platform: achieving commercial sustainability

One of the summit’s defining themes is how genome editing programs can scale beyond rare, single-patient applications toward commercially viable, multi-indication platforms. A fireside chat entitled Transitioning gene editing therapeutics from bespoke treatments to platform approaches for broader disease indications and commercial sustainability will bring together Birgit Schultes (Chief Scientific Officer, Intellia Therapeutics), Wenning Qin (Senior Vice President, eGenesis), Devyn Smith (Chief Executive Officer, Arbor Biotechnologies), and Alan Brooks (Senior Vice President, Research, Metagenomi Therapeutics) to examine the strategic, regulatory, and technical barriers currently limiting the field, and the platform design decisions required to transition toward scalable, multi-disease treatment models.

Safety, off-targeting, and clinical translation

Establishing robust safety profiles and translating preclinical data into meaningful clinical outcomes are priorities running throughout the program. Gopi Shanker (Chief Scientific Officer, Beam Therapeutics), who chairs Day 1, will present on Translating genetic insights by optimizing sickle cell therapies from bench to bedside to achieve clinical success, examining how programs anchored in the critical 60/40 hemoglobin ratio can be aligned with target product profiles to reduce vaso-occlusive crises to registrational endpoints. Complementing this, Ishani Dasgupta (Senior Scientist, AbbVie) will address how expanded CRISPR arrayed screens can be designed and deployed to identify and validate novel targets within early-stage discovery for autoimmune diseases.

Delivery innovation: AAV, non-viral, and extrahepatic approaches

Expanding the reach of gene editing beyond the liver is one of the field’s most pressing technical challenges, and the 2026 program gives significant attention to delivery innovation across viral and non-viral platforms. Kyle Watters (Director, Precision Editing, Arbor Biotechnologies) will present on packaging prime and RT editing technologies within AAV despite significant size constraints, and how this compares to conventional CRISPR for next-generation in vivo editing. The pre-conference workshop day will feature a complementary session on extrahepatic delivery, led by Bin Wu (Founder and Chief Executive Officer, Cytodigm), covering approaches to reducing LNP and AAV liver tropism, enhancing tissue-specific targeting, and investigating exosomal delivery across cardiovascular, neurological, and other disease areas.

Manufacturing, CMC, and CDMO collaboration

Scalable manufacturing and strong CMC strategy are essential to realizing the commercial promise of genome editing. A panel discussion moderated by Gopi Shanker will examine these challenges alongside Wei-Chiang Chen (Associate Director, Sanofi) and Cindy Liu (Director, Molecular Analytical Development, Prime Medicine). Key topics include preserving product purity and potency throughout scale-up, optimizing CDMO partnerships and tech transfer, and developing flexible guide RNA manufacturing approaches to support production across diverse rare disease populations.

Regulatory strategy and IND readiness

Regulatory preparedness is a thread running throughout all three days. The pre-conference workshop will open with a session on navigating IND packages for novel genome editing modalities, led by John Moore (Senior Director, Translational Gene Editing, Eli Lilly) and Lauren Young (Senior Director, Genomics and Computational Biology, Beam Therapeutics), covering submission strategy, preclinical-to-clinical bridging, and direct agency engagement. A roundtable moderated by Jesse Owens (Chief Scientific Advisor, Komo Biosciences) will explore how to build regulatory confidence in emerging modalities, including prime editing, base editing, and epigenetic editing.

Across three focused days, the 7th Annual Genome Editing Therapeutics Summit will offer an applied forum for the genome editing community to address the translational, manufacturing, and regulatory challenges standing between scientific innovation and patient impact. Bringing together preclinical, clinical, regulatory, and commercial leaders, the summit represents a key opportunity to define the practical steps from bench to bedside.

As a reader of Cell and Gene Therapy Insights, you are entitled to a 10% discount on delegate tickets – just use the code CGTI7190810 at checkout. You can find out more about the event here.

Additionally, to find out what other cell and gene therapy events are upcoming, you can find our online Events Calendar here.